A More Precise Gene Editor Takes Aim at Cystic Fibrosis Mutations
A redesigned base-pair gene editor developed at the University of Pennsylvania and Rice University cuts unintended 'bystander' DNA mutations by more than 80% while preserving editing activity at the intended target. Published in Molecular Therapy, the work targets a class of mutations common in cystic fibrosis involving clustered cytosine bases -- a pattern found in three-quarters of the mutations this editor type can address. The research is preclinical but represents a meaningful advance in gene-editing precision.